Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease

Krabbe disease is a rare neurodegenerative disorder caused by a deficiency in galactocerebrosidase. The only effective treatment is hematopoietic stem cell transplantation (HSCT). Approximately 85% of Krabbe disease cases are the infantile subtypes, among which ∼20% are late infantile. Prior studies...

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Vydáno v:Blood
Hlavní autoři: Yoon, Isabel C., Bascou, Nicholas A., Poe, Michele D., Szabolcs, Paul, Escolar, Maria L.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Hematology 2021
Témata:
Clinical Trials and Observations
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8020262/
https://ncbi.nlm.nih.gov/pubmed/33150395
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood.2020005477
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